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FDA Approves First Gene Therapy For Leukemia
The Food and Drug Administration on Wednesday announced what the agency calls a "historic action" — the first approval of a cell-based gene therapy in the United States.
The FDA approved Kymriah, which scientists refer to as a "living drug" because it involves using genetically modified immune cells from patients to attack their cancer.
The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It's also known as CAR-T cell therapy.
The treatment, which is also called CTL019, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July.
The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.
Novartis, which developed the drug, says the one-time treatment will cost $475,000 for patients who respond. People who do not respond within a month would not be charged, and the company said it is taking additional steps to make sure everyone who needs the drug can afford it.
The Food and Drug Administration on Wednesday announced what the agency calls a "historic action" — the first approval of a cell-based gene therapy in the United States.
The FDA approved Kymriah, which scientists refer to as a "living drug" because it involves using genetically modified immune cells from patients to attack their cancer.
The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It's also known as CAR-T cell therapy.
The treatment, which is also called CTL019, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July.
The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.
Novartis, which developed the drug, says the one-time treatment will cost $475,000 for patients who respond. People who do not respond within a month would not be charged, and the company said it is taking additional steps to make sure everyone who needs the drug can afford it.
